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Desmopressin responsiveness by age in type 1 von Willebrand disease

机译:von Willebrand疾病1型von von Willebrand疾病的年龄反应性

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Background Patients with type 1 von Willebrand disease (VWD) undergo a desmopressin (DDAVP) responsiveness challenge at diagnosis to assess whether DDAVP reverses their coagulation deficits. Current practice assumes DDAVP responsiveness remains constant over the lifetime. In patients with type 1 VWD, VWF‐related parameters increase with age. This study explores whether DDAVP responsiveness also differs with age in this population. Methods We conducted a retrospective chart review of 106 patients enrolled at our center since 1990. Our primary outcome was DDAVP responsiveness at 1?hour after DDAVP challenge. Locally weighted scatterplot smoothing fit and Spearman correlation coefficients were used to study the relationship between age and DDAVP responsiveness. For female participants, we used the Kruskal‐Wallis test to compare absolute and relative changes in DDAVP responsiveness at various ages. Results We had 79 patients (56 female) with type 1 VWD with at least 1 DDAVP challenge. In women with type 1 VWD, the absolute change in DDAVP responsiveness did not vary significantly with age (VWF:antigen [Ag], ?0.08, P =?.56; VWF:ristocetin cofactor [RCo], ?0.16, P =?.26; low‐molecular‐weight component of factor VIII [FVIII:C], ?0.01, P =?.93), nor did the relative change in DDAVP responsiveness (VWF:Ag, ?0.03, P =?.86; VWF:RCo, ?0.25, P =?.09; FVIII:C, ?0.14, P =?.34). The data plot suggested a relationship. Conclusion In women with type 1 VWD, DDAVP responsiveness may vary over the life cycle. Our exploratory findings are limited by our retrospective data, cross‐sectional design, and small sample. Future studies should investigate the relationship between age and DDAVP responsiveness prospectively to evaluate whether there is clinical utility in rechallenging postpubertal female patients with type 1 VWD.
机译:背景技术患者1型von willebrand疾病(vwd)在诊断中经历了去载素(DDAVP)响应性挑战,以评估DDAVP是否逆转其凝固赤字。目前的实践假设DDAVP响应能力在寿命中保持不变。在1型VWD患者中,VWF相关参数随着年龄的增长而增加。本研究探讨了DDAVP响应性也与该人群年龄不同。方法对自1990年以来,我们对106名患者进行了回顾性图表综述。我们的主要结果是DDAVP挑战后1?小时的DDAVP响应性。用于局部加权散点图平滑拟合和Spearman相关系数来研究年龄与DDAVP响应性之间的关系。对于女性参与者,我们使用了Kruskal-Wallis测试来比较各个年龄段的DDAVP响应性中的绝对和相对变化。结果我们有79名患者(56名女性),型号为1 VWD,至少有1名DDAVP挑战。在患有1型VWD的女性中,年龄(VWF:抗原[Ag],Δ0.08,p =Δ.,vwf:risocetin cofactor [rco],?0.16,p =? .26;因子VIII [FVIII]的低分子量分量[FVIII:C],?0.01,P =β.93),也不是DDAVP响应性的相对变化(VWF:AG,?0.03,P =Δ.86; VWF:RCO,?0.25,P =?09; FVIII:C,?0.14,P =?34)。数据绘图建议了一种关系。结论在1型VWD的女性中,DDAVP响应能力可能会在生命周期内变化。我们的探索性调查结果受我们的回顾性数据,横截面设计和小样本的限制。未来的研究应该调查年龄和DDAVP对响应性之间的关系,促使响应性,评估是否有临床公用事业在重新培养型女性患者1型VWD中。

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